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A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
Sponsor: BioMarin Pharmaceutical
Summary
This is a Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia. The primary objective is to evaluate the long-term safety and tolerability of daily SC injections of BMN 111 in children with ACH who have completed two years of treatment in the 111-202 study.
Official title: A Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children With Achondroplasia
Key Details
Gender
All
Age Range
7 Years - Any
Study Type
INTERVENTIONAL
Enrollment
30
Start Date
2016-01-26
Completion Date
2028-02
Last Updated
2026-03-13
Healthy Volunteers
No
Conditions
Interventions
BMN 111
BMN 111 will be administered subcutaneously daily.
Locations (9)
Children's Hospital & Research Center Oakland
Oakland, California, United States
Harbor - UCLA Medical Center
Torrance, California, United States
Ann and Robert H. Lurie Childrens Hospital of Chicago
Chicago, Illinois, United States
Johns Hopkins McKusick - Institute of Genetic Medicine
Baltimore, Maryland, United States
Vanderbilt University
Nashville, Tennessee, United States
Baylor College of Medicine
Houston, Texas, United States
Murdoch Children's Research Institute
Parkville, Victoria, Australia
Institut Necker
Paris, France
Guys & St. Thomas NHS Foundation Trust Evelina Hospital
London, United Kingdom